A Study of JR-141 in Patients With Mucopolysaccharidosis II

• Register: ClinicalTrials.gov
• Last refreshed on: 21 November 2022
• Main ID: NCT03568175
• Date of registration: 01/06/2018
• Prospective Registration: Yes
• Primary sponsor: JCR Pharmaceuticals Co., Ltd.
• Public title: A Study of JR-141 in Patients With Mucopolysaccharidosis II
• Scientific title: A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
• Date of first enrolment: August 1, 2018
• Target sample size: 28
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03568175
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Japan

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Capable of providing written consent by himself (not mandatory for those aged under 20
years at the time of informed consent process, or those who is impossible to obtain
consent from the patient himself due to intellectual disabilities associated with MPS
II.)

2. In the case of a patient who is under the age of 20 years or from whom it is not
possible to obtain consent due to intellectual disabilities associated with MPS II, he
may be included if written consent can be provided by legal representative (however,
written consent should be obtained from the patient himself too, whenever possible)

3. Males with confirmed diagnosis of MPS II, based on deficient activity of
iduronate-2-sulfatase (IDS) in leucocytes, plasma or fibroblasts and/or pathogenic
mutations identified in the IDS gene, etc.

4. Naïve patients or patients who are receiving stable enzyme replacement therapy with
Elaprase for more than 8 weeks before the observational period starts.

Exclusion Criteria:

1. Previous engrafted HSCT, excluding those who need enzyme replacement therapy even
after HSCT.

2. Judged by the investigator or subinvestigator as being unable to undergo lumbar
puncture, including those who have difficulties in taking position for lumbar puncture
due to joint contracture or those who are likely experience difficulty breathing
during the lumbar puncture process.

3. Judged by the investigator or subinvestigator to be ineligible to participate in the
study due to a history of serious drug allergy or sensitivity.

4. Patients who have received other investigational product within 4 months before
enrollment in the study.

5. Otherwise judged by the investigator or subinvestigator to be ineligible to
participate in the study.



Age minimum: N/A
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis II

Intervention(s)

Drug: JR-141

Primary Outcome(s)

Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid [Time Frame: Baseline to 52 weeks]

Secondary Outcome(s)

Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001) [Time Frame: 25, 52 weeks]

Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid. [Time Frame: Baseline to 25 weeks]

Change From Baseline in 6-minute Walk Test Distance. [Time Frame: Baseline, 25 weeks, 52 weeks]

Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. ) [Time Frame: 25, 52 weeks]

Change From Baseline in Cardiac Function. [Time Frame: Baseline, 25 weeks, 52 weeks]

Change From Baseline in Joint Range of Motion. [Time Frame: Baseline, 25 weeks, 52 weeks]

Change From Baseline in Liver Volumes. [Time Frame: Baseline, 25 weeks, 52 weeks]

Change From Baseline in Urinary Heparan Sulfate Levels. [Time Frame: Baseline, 25 weeks, 52 weeks]

Drug concentration in Cerebrospinal Fluid. [Time Frame: 25, 52*weeks]

Change From Baseline in Serum Heparan Sulfate Levels. [Time Frame: Baseline, 24-26 weeks, 50-52 weeks]

Change From Baseline in Spleen Volumes. [Time Frame: Baseline, 25 weeks, 52 weeks]

Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid. [Time Frame: Baseline, 25 weeks, 52 weeks]

Change From Baseline in Serum Dermatan Sulfate Levels. [Time Frame: Baseline, 24-26 weeks, 50-52 weeks]

Change From Baseline in Urinary Dermatan Sulfate Levels. [Time Frame: Baseline, 25 weeks, 52 weeks]

Secondary ID(s)

JR-141-301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors